The objectives of this time-to-event study were to assess the efficacy and safety of eculizumab as compared with placebo in participants with neuromyelitis optica spectrum disorder (NMOSD) who were anti-aquaporin-4 (AQP4) antibody-positive.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
143
Induction Phase: 900 mg IV weekly for 4 weeks, followed by 1200 mg for the fifth dose; Maintenance Phase: 1200 mg IV every 2 weeks
Induction Phase: matching placebo (900 mg) IV weekly for 4 weeks, followed by matching placebo (1200 mg) for the fifth dose; Maintenance Phase: matching placebo (1200 mg) IV every 2 weeks
Participants With An Adjudicated On-trial Relapse
An On-trial Relapse was defined as a new onset of neurologic symptoms or worsening of existing neurologic symptoms with an objective change (clinical sign) on neurologic examination that persisted for more than 24 hours as confirmed by the treating physician. An adjudicated On-trial Relapse was defined by the protocol and positively adjudicated by the relapse adjudication committee.
Time frame: Baseline, Up To 211 Weeks (End of Study)
Adjudicated On-trial Annualized Relapse Rate (ARR)
The adjudicated On-trial ARR was computed as the total number of relapses divided by the total number of patient years in the study period. A central independent committee was used to adjudicate all On-trial Relapses as determined by the treating physician. Results reported as adjusted adjudicated On-trial ARR based on a Poisson regression adjusted for randomization strata and historical ARR in 24 months prior to Screening.
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In EDSS At End Of Study
Disease-related disability was measured by the EDSS. The EDSS is an ordinal clinical rating scale that ranges from 0 (normal neurologic examination) to 10 (death) in half-point increments. A decrease in score indicates improvement.
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In Modified Rankin Scale (mRS) Score At End Of Study
Disease-related disability was measured by the mRS score. The mRS is a commonly used scale for measuring the degree of disability or dependence in the daily activities of people who have suffered from a neurological disability. The scale ranges from 0 (no disability) to 6 (death) in whole-point increments. A decrease in score indicates improvement.
Time frame: Baseline, Up To 211 Weeks (End of Study)
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Mayo Clinic Arizona
Scottsdale, Arizona, United States
The Research Center of Southern California
Carlsbad, California, United States
Georgetown University Hospital
Washington D.C., District of Columbia, United States
University of Miami McKnight Brain Institute
Miami, Florida, United States
Neurological Services of Orlando
Orlando, Florida, United States
Fort Wayne Neurological Center
Fort Wayne, Indiana, United States
University of Kansas Medical Center
Kansas City, Kansas, United States
Baptist Health Lexington
Lexington, Kentucky, United States
University of Maryland Medical Center
Baltimore, Maryland, United States
John Hopkins University School of Medicine
Baltimore, Maryland, United States
...and 60 more locations
Change From Baseline In Hauser Ambulation Index (HAI) Score At End of Study
The HAI evaluates gait and was used to assess the time and effort used by the participant to walk 25 feet (8 meters). The scale ranges from 0 to 9, with 0 being the best score (asymptomatic; fully ambulatory with no assistance) and 9 being the worst (restricted to wheel chair; unable to transfer self independently). A decrease in score indicates improvement.
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In European Quality Of Life (EuroQoL) Health 5-Dimension Questionnaire (EQ-5D) Visual Analogue Scale At End Of Study
The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Assessments were made using the EQ-5D Visual Analogue Scale, which captures the self-rating of current health status using a visual "thermometer" with the endpoints of 100 (best imaginable health state) at the top and zero (worst imaginable health state) at the bottom. An increase in score indicates improvement.
Time frame: Baseline, Up To 211 Weeks (End of Study)
Change From Baseline In EuroQoL EQ-5D Index Score At End Of Study
The EuroQoL EQ-5D is a generic, standardized, self-administered instrument that provides a simple, descriptive profile and a single index value for health status. Index scores range from less than 0 to 1, with higher scores representing a better health status.
Time frame: Baseline, Up To 211 Weeks (End of Study)